PKU Patient Advocates for Coverage of Treatment for All Ages in the UK

According to a story from, a treatment for phenylketonuria (PKU) is currently under consideration for coverage on the UK’s National Health Service (NHS). The treatment is called Kuvan and is manufactured by the US drug company BioMarin. A current proposal is being floated that would allow coverage of the drug, but only for patients under age 18. However, PKU patient Lily Wood, along with 240 others, has been speaking out and calling for coverage for patients of all ages. 

About Phenylketonuria (PKU)

PKU is a type of metabolic disorder affecting the enzyme phenylalanine, which builds up in the body and can cause serious problems. Normally, phenylalanine is broken down, but a mutation affecting the PAH gene prevents this from happening in patients with the disorder. Prompt treatment is essential to avoid major complications. Symptoms of PKU include small head size, low birth weight, an unusual musty odor, pale skin, behavioral problems, intellectual disability, heart issues, seizures, and mental disorders. All of these symptoms can be prevented with proper and timely treatment; PKU is routinely screened for in newborns for this reason. The primary management strategy is a highly controlled diet that is low in phenylalanine. Supplements and enzyme substitutes may also be considered. Most patients eventually go off the diet when they reach adulthood. Some older patients may still have trouble managing the condition later in life. To learn more about PKU, click here.

Calling for Coverage for All Patients

Lily is 19 years old and would not have access to the treatment under the latest proposal. She penned a letter to Health Secretary Matt Hancock criticizing the plan as unethical and would only be used to save funding by avoiding supporting older patients:

“It is vital Kuvan is accessible to all PKU sufferers – as the improvement to quality of life would be immense.” – Lily Wood

Lily’s point holds true as the diet that PKU patients must follow in absence of treatment severely limits their options when it comes to food and nutrition. In a sense, these patients are forced to sit out the enjoyment of food that unaffected people experience—or suffer dire consequences.

Health minister Lord Bethell has implied that the plan may be subject to change, leaving open the possibility that coverage could be extended to patients of all ages. The National Institute for Health and Care Excellence (NICE) is scheduled to release its final guidance regarding Kuvan in June.

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